The Years in Which Gene Therapy Finally Escapes the Labs and Trials

The costs of biotechnologies relating to gene therapy and genetic analysis have fallen steeply in the last ten years, even as the capabilities of a well-equipped laboratory have increased by leaps and bounds over the same period of time. A graduate student today has more power at his or her fingertips than an entire laboratory staff of the early 90s. This has matched the economics of computing hardware, as much of biotechnology is essentially a matter of building direct interfaces between that computing hardware and the nanoscale life science world of proteins and cells. This said, until just the past year or two the available options for gene therapy and most genetic analysis remained still remained too challenging and expensive for growth into the market. They have long been practical for the work of a laboratory or research institution, but not for most potential uses in the mass market, where a single provider would be expected to churn through thousands or tens of thousands of samples in a week, with high reliability and at a minimal expense per item.

The present leap in capacity and fall in cost promises to change all of that, however, given time to work its way through the pipeline. The latest methods and technologies are so far proving to be cheap enough and reliable enough to form the basis for the mass commercialization of genetic analysis and alteration in the years ahead. This will certainly have a great impact on many areas of medicine, though we'll probably all be surprised by many of the specific outcomes. The best thing that can happen for progress in the long term is for the cost of research to fall greatly, as is presently happening. The lower the cost of entry to a field, the more experimentation and development that will take place - and this is why it is helpful to keep an eye on progress in fundamental technologies, not just on specific applications of interest, such as in the area of aging and longevity.

New DNA-editing technology spawns bold UC initiative

The technology, precision "DNA scissors" referred to as CRISPR/Cas9, has exploded in popularity since it was first published in June 2012 and is at the heart of at least three start-ups and several heavily-attended international meetings. Scientists have referred to it as the "holy grail" of genetic engineering. "The CRISPR/Cas9 technology is a complete game changer. With CRISPR, we can now turn genes off or on at will."

The new genomic engineering technology significantly cuts down the time it takes researchers to test new therapies. CRISPR/Cas 9 allows the creation in weeks rather than years of animal strains that mimic a human disease, allowing researchers to test new therapies. The technique also makes it quick and easy to knock out genes in human cells or in animals to determine their function, which will speed the identification of new drug targets for diseases.

Using the Cas9 technique, UC Berkeley immunologist Russell Vance disabled a gene in mice that regulates fur color and in just six weeks had a strain of mice with white coats instead of brown. Similar research in animal models ranging from rodents to primates is being done in labs around the world using the CRISPR/Cas9 technology. Other researchers have already adapted the technology to reprogram stem cells to regenerate damaged organs, such as the liver, and made attempts to reprogram immune cells to cure AIDS in HIV-positive patients.

Innovative technique provides inexpensive, rapid and detailed analysis of proteins

Mass Spectrometric Immunoassay (MSIA) [is] a high-throughput protein quantification technique that also provides detailed protein information. In a new study [researchers] demonstrate the power of the MSIA platform, with a vision towards clinical adoption. The research reports a high-throughput method for quantifying and characterizing insulin-like growth factor 1 (or IGF1) at a rate of more than 1,000 human samples a day.

Mass spectroscopy can readily identify genetic variants that are expressed on the protein level (for example single-nucleotide polymorphisms). Such changes may alter or disable the function of the resulting protein. Further, mass spectroscopy can pinpoint changes that may occur to the protein after it has already been produced from the gene template - so-called post-translational modifications.